用于基因治疗的细胞外囊泡

IF 37.6
Emilio Di Ianni, Wataru Obuchi, Koen Breyne, Xandra O. Breakefield
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引用次数: 0

摘要

基因疗法为治疗以前无法治愈的疾病带来了希望,如遗传性疾病、癌症和自身免疫性疾病。然而,基因治疗需要具有细胞和组织特异性的有效递送,由于病毒递送载体的靶向性和载货能力有限,以及免疫原性和毒性问题,这仍然具有挑战性。细胞外囊泡可以被设计成基因治疗的非病毒载体,因为它们能够运送多种货物类型,包括转基因、小编码或非编码RNA、DNA和功能蛋白。重要的是,细胞外囊泡是免疫中性的,可以跨越生物屏障。本文就细胞外囊泡在基因治疗中的应用作一综述。我们概述了细胞外囊泡的固有内容如何促进不同的基因治疗方法,并研究了细胞外囊泡的设计,以装载基因治疗工具,靶向递送和货物释放。最后,我们调查了细胞外囊泡的临床应用,并强调了重要的工程和转化挑战。细胞外囊泡可以被设计成基因治疗的传递平台。本文讨论了细胞外囊泡在不同基因治疗方式中的应用和工程,概述了将该技术推向临床的关键步骤。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Extracellular vesicles for the delivery of gene therapy

Extracellular vesicles for the delivery of gene therapy
Gene therapy has brought hope for the treatment of previously incurable diseases, such as genetic disorders, cancers and autoimmune diseases. However, gene therapy requires efficient delivery with cell and tissue specificity, which remains challenging owing to the limited targeting and cargo-loading capacity of viral delivery vehicles, as well as immunogenicity and toxicity concerns. Extracellular vesicles can be designed as non-viral carriers for gene therapy owing to their ability to deliver multiple cargo types, including transgenes, small encoding or non-coding RNA, DNA and functional proteins. Importantly, extracellular vesicles are immunologically neutral and can cross biological barriers. In this Review, we discuss the application of extracellular vesicles in gene therapy. We outline how the inherent content of extracellular vesicles can facilitate different gene-therapy approaches and examine the design of extracellular vesicles for the loading of gene-therapy tools, targeted delivery and cargo release. Finally, we survey clinical applications of extracellular vesicles and highlight important engineering and translational challenges. Extracellular vesicles can be designed to serve as delivery platforms for gene therapy. This Review discusses the application and engineering of extracellular vesicles for different gene-therapy modalities, outlining crucial steps to advance this technology into the clinic.
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